The first half of 2019 was filled with denial after realizing something was wrong with Raghav’s health. Aug 2019 brought a change. Raghav’s diagnosis in August gave a dose of reality and redefined our hopes, dreams and goals. Raghav was diagnosed with a rare genetic mutation in a gene called GPX4. In simple english, Raghav’s body lacks the basic energy required to get through his daily activities. 

Since then our focus has been on making Raghav independent, successful and happy in however he sees the world to be. We are working on changing the traditional drug development pipeline to speed up the process of finding a drug for Raghav and kids affected with other N-of-1 rare conditions. 

Research progress in 2019

• We put together a phenomenal team of researchers and scientific advisors who are the best minds in the world to find a cure for Raghav’s condition

• We are creating a collaboration network of researchers working on this condition to freely share data, ideas, protocols, materials and results.

• We came up with roadmap explaining our approach to drug discovery. We will continue to collaborate with the scientific team to publish this paper in 2020

• To facilitate broader research, we are working on creating and phenotyping animal models, creating iPSC cells and controls publicly available for any scientist who wants to contribute to the research

• Going through several research papers, we came up with a list of drugs that can potentially be repurposed to address Raghav’s condition. Based on the above analysis, we are ready to file an FDA Expanded Access petition for an experimental drug. If everything goes well, we will start Raghav on this drug in Feb 2020.

• We have started experiments to understand the basic science behind the mutation and how it affects Raghav. While this can traditionally takes years, we are actively managing and thinking ahead to keep moving this forward.

• We are organizing a “CureGPX4 Research Conference” in March 2020 to bring all the scientists together to share research progress and collaborate on finding a faster cure.

• We found 3 other kids affected by this condition. We will rally together to find a cure for every baby affected with this genetic condition.

None of this would have been possible without the amazing support of family, friends and advisors. You guys continue to stand by us while we march towards making Raghav a stronger kiddo! 

2020, here we come!

let us see what you have in store for us